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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT00847379




Registration number
NCT00847379
Ethics application status
Date submitted
16/02/2009
Date registered
19/02/2009

Titles & IDs
Public title
Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)
Scientific title
A Phase 2B Extension Study of PTC124 in Subjects With Nonsense-Mutation-Mediated Duchenne and Becker Muscular Dystrophy
Secondary ID [1] 0 0
PTC124-GD-007e-DMD
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Duchenne Muscular Dystrophy 0 0
Becker Muscular Dystrophy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Ataluren

Experimental: Overall Participants: High-Dose Ataluren - All participants will receive ataluren suspension orally three times a day (TID), 20 mg/kg at morning, 20 mg/kg at midday, and 40 mg/kg at evening (total daily dose 80 mg/kg) for up to 96 weeks in this study. Any participant who was receiving a reduced dose of ataluren at the end of treatment visit in study PTC124-GD-007-DMD, will be initiated ataluren therapy in this extension study at the 5-, 5-, and 10-mg/kg dose level; dose will be increased to 10, 10, and 20 mg/kg at Week 6 and to 20, 20, and 40 mg/kg at Week 12, if the preceding dose level is well tolerated.


Treatment: Drugs: Ataluren
Ataluren oral powder for suspension will be administered as per dose and schedule specified in the arm.
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Treatment-Emergent Adverse Events (AEs)
Timepoint [1] 0 0
Baseline (Week 48 of Study 007) up to Week 102
Primary outcome [2] 0 0
Number of Participants With Clinically Significant Abnormal Laboratory Parameters
Timepoint [2] 0 0
Baseline (Week 48 of Study 007) up to Week 102
Secondary outcome [1] 0 0
Change From Baseline in 6-Minute Walk Distance (6MWD) at Week 60
Timepoint [1] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [2] 0 0
Change From Baseline in Mean Activity Period/Day/Visit at Week 60, as Assessed by Step Activity Monitoring (SAM)
Timepoint [2] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [3] 0 0
Change From Baseline in Mean Total Step Count/Day/Visit During the Active Periods at Week 60, as Assessed by SAM
Timepoint [3] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [4] 0 0
Change From Baseline in Mean Total Step Count/Hour During the Active Period at Week 60, as Assessed by SAM
Timepoint [4] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [5] 0 0
Change From Baseline in Maximum Continuous 10-minute, 20-minute, 30-minute, and 60-minute Total Step Count at Week 60, as Assessed by SAM
Timepoint [5] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [6] 0 0
Change From Baseline in Percentage of Time During Active Period Spent at No Activity (0 Steps/Minute[Min]), Low Activity (Less Than or Equal to [=]15 Steps/Min), Medium Activity (16-30 Steps/Min), and High Activity (Greater Than[>]30 Steps/Min) at Week 60
Timepoint [6] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [7] 0 0
Change From Baseline in Time to Stand From Supine Position at Week 60
Timepoint [7] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [8] 0 0
Change From Baseline in Time to Walk/Run 10 Meters at Week 60
Timepoint [8] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [9] 0 0
Change From Baseline in Time to Climb 4 Stairs at Week 60
Timepoint [9] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [10] 0 0
Change From Baseline in Time to Descend 4 Stairs at Week 60
Timepoint [10] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [11] 0 0
Change From Baseline in Heart Rate Before, During, and After Each 6MWD Test at Week 60, as Assessed by Heart Rate Monitoring With the Polar® RS400
Timepoint [11] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [12] 0 0
Change From Baseline in Number of Digits Recalled Forwards and Backwards on Digit Span Task at Week 60
Timepoint [12] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [13] 0 0
Change From Baseline in Participant- Reported Health-Related Quality of Life (HRQL) as Measured by the Pediatric Quality of Life Inventory (PedsQL) Physical, Emotional, Social, and School Functioning Domain Scores at Week 60
Timepoint [13] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [14] 0 0
Change From Baseline in Parent/Caregiver- Reported HRQL as Measured by the PedsQL Physical, Emotional, Social, and School Functioning Domain Scores at Week 60
Timepoint [14] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [15] 0 0
Change From Baseline in Participant-Reported HRQL as Measured by the Total Fatigue Scale Score at Week 60
Timepoint [15] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [16] 0 0
Change From Baseline in Parent/Caregiver-Reported HRQL as Measured by the Total Fatigue Scale Score at Week 60
Timepoint [16] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [17] 0 0
Change From Baseline in Participant and Parent/Caregiver Reported Activities of Daily Living of Participants Who Were Unable to Complete the 6MWT (Nonambulatory Participants), as Measured by the Egen Klassifikation (EK) Scale at Week 60
Timepoint [17] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [18] 0 0
Change From Baseline in Parent/Caregiver-Reported Treatment Satisfaction Questionnaire for Medication (TSQM) Score at Week 60
Timepoint [18] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [19] 0 0
Change From Baseline in Serum Concentration of Creatine Kinase (CK) at Week 60
Timepoint [19] 0 0
Baseline (Week 48 of Study 007), Week 60
Secondary outcome [20] 0 0
Study Drug Compliance
Timepoint [20] 0 0
Baseline (Week 48 of Study 007) to Week 96
Secondary outcome [21] 0 0
Trough Ataluren Plasma Concentration
Timepoint [21] 0 0
Pre-morning dose (0 hour) at Baseline (Week 48 of 007 study), Weeks 54, 60, 72, 84, and 96

Eligibility
Key inclusion criteria
* Completion of blinded study drug treatment in the previous Phase 2b study (PTC124-GD-007-DMD).
* Ability to provide written informed consent (parental/guardian consent if applicable)/assent (if less than [<]18 years of age).
* In participants who are sexually active, willingness to abstain from sexual intercourse or employ a barrier or medical method of contraception during PTC124 administration and the 6-week follow up period.
* Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, laboratory tests, and study restrictions.
Minimum age
5 Years
Maximum age
No limit
Sex
Males
Can healthy volunteers participate?
No
Key exclusion criteria
* Known hypersensitivity to any of the ingredients or excipients of the study drug (Litesse® UltraTM [refined polydextrose], polyethylene glycol 3350, Lutrol® micro F127 [poloxamer 407], mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, Cab-O-Sil® M5P [colloidal silica], magnesium stearate).
* Ongoing participation in any other therapeutic clinical trial.
* Prior or ongoing medical condition (for example, concomitant illness, psychiatric condition, behavioral disorder, alcoholism, drug abuse), medical history, physical findings, ECG findings, or laboratory abnormality that, in the investigator's opinion, could adversely affect the safety of the subject, makes it unlikely that the course of treatment or follow up would be completed, or could impair the assessment of study results.

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
31/01/2009
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
24/05/2010
Sample size
Target
Accrual to date
Final
173
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
The Royal Children's Hospital - Parkville
Recruitment hospital [2] 0 0
The Children's Hospital at Westmead - Westmead
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment postcode(s) [2] 0 0
2145 - Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Florida
Country [4] 0 0
United States of America
State/province [4] 0 0
Iowa
Country [5] 0 0
United States of America
State/province [5] 0 0
Kansas
Country [6] 0 0
United States of America
State/province [6] 0 0
Massachusetts
Country [7] 0 0
United States of America
State/province [7] 0 0
Minnesota
Country [8] 0 0
United States of America
State/province [8] 0 0
Missouri
Country [9] 0 0
United States of America
State/province [9] 0 0
New York
Country [10] 0 0
United States of America
State/province [10] 0 0
North Carolina
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
Oregon
Country [13] 0 0
United States of America
State/province [13] 0 0
Pennsylvania
Country [14] 0 0
United States of America
State/province [14] 0 0
Texas
Country [15] 0 0
United States of America
State/province [15] 0 0
Utah
Country [16] 0 0
Belgium
State/province [16] 0 0
Leuven
Country [17] 0 0
Canada
State/province [17] 0 0
Ontario
Country [18] 0 0
Canada
State/province [18] 0 0
Vancouver
Country [19] 0 0
France
State/province [19] 0 0
Marseille cedex 20
Country [20] 0 0
France
State/province [20] 0 0
Nantes cedex 1
Country [21] 0 0
France
State/province [21] 0 0
Paris
Country [22] 0 0
Germany
State/province [22] 0 0
Essen
Country [23] 0 0
Germany
State/province [23] 0 0
Freiburg
Country [24] 0 0
Israel
State/province [24] 0 0
Jerusalem
Country [25] 0 0
Italy
State/province [25] 0 0
Milano
Country [26] 0 0
Italy
State/province [26] 0 0
Roma
Country [27] 0 0
Spain
State/province [27] 0 0
Barcelona
Country [28] 0 0
Spain
State/province [28] 0 0
Valencia
Country [29] 0 0
Sweden
State/province [29] 0 0
Goteborg
Country [30] 0 0
Sweden
State/province [30] 0 0
Stockholm
Country [31] 0 0
United Kingdom
State/province [31] 0 0
London
Country [32] 0 0
United Kingdom
State/province [32] 0 0
Newcastle Upon Tyne
Country [33] 0 0
United Kingdom
State/province [33] 0 0
Oswestry

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
PTC Therapeutics
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Genzyme, a Sanofi Company
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Leone Atkinson, M.D., Ph.D.
Address 0 0
PTC Therapeutics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT00847379